Hetrombopag plus caffeic acid tablets for the treatment of chemotherapy-induced thrombocytopenia in solid tumors: A single-center, open-label, phase II trial.

12130 Background: Chemotherapy-induced thrombocytopenia (CIT) frequently complicates cancer therapy, resulting in treatment delays, dose reductions, and premature discontinuation. However, no widely approved therapies are currently available to address this clinical gap. This phase II trial was designed to evaluate the efficacy and safety of the thrombopoietin receptor agonist hetrombopag combined with caffeic acid tablets in this patient population. Methods: This is a single-arm, single-center, prospective phase II trial (ChiCTR2400086043) designed to enroll patients with malignancies experiencing thrombocytopenia (platelet count ≤ 75 ×10⁹/L) during anti-cancer therapy. Eligible participants received hetrombopag 5 mg once daily (QD) plus caffeic acid tablets 0.3 g three times daily (TID) for 14 consecutive days. The primary endpoint is the proportion of patients achieving a platelet count ≥100×10⁹/L after treatment; secondary endpoints included time to first response (platelet count ≥100×10⁹/L), proportion of patients with platelet recovery to ≥ 75×10⁹/L, and safety profile of the regimen. Results: A total of 65 patients were enrolled between 24 June 2024 and 25 December 2025.The median age of enrolled patients was 60.0 years, and 70.8% of patients had a baseline platelet count of ≥50×10⁹/L. The most common tumor types were colorectal cancer (23.1%) and gastric cancer (13.8%). The majority (41.5%) received oxaliplatin-based chemotherapy, 26.2% received immunotherapy plus chemotherapy, and 18.5% received targeted therapy plus chemotherapy. After treatment with hetrombopag plus caffeic acid tablets, 84.6% (55/65) of patients achieved platelet counts ≥100×10⁹/L and 87.7% (57/65) achieved ≥75×10⁹/L. The median durations from the first dose of hetrombopag plus caffeic acid tablets platelet response ≥100×10⁹/L was 7 days (range:5–9). Adverse drug reactions were reported in 17 patients; no drug-related adverse events of grade ≥3 occurred. Conclusions: Hetrombopag plus caffeic acid tablets confer outstanding therapeutic efficacy in CIT while maintaining a well-tolerated safety profile. This oral regimen fulfills an unmet clinical need and provides a practical, non-invasive therapeutic option for CIT management. Clinical trial information: ChiCTR2400086043.